“What can I do to cure leukemia? I’ll try anything,” he said to me seriously. “I heard you’re doing research here. I want to join!”
He was interested in participating in a clinical trial of a new immunotherapy that uses the patient’s own immune system to target and eliminate leukemic cells. His doctor told him the trial was available at our cancer center.
Clinical trials are phase 1 trials, more dramatically called “first in human” trials. This is the first time the drug has been administered to people following what is called a “preclinical” trial. The main objectives of phase 1 trials are to find the appropriate drug dosage and evaluate the drug’s safety. No Determine if a drug really works.
In fact, these trials suggest that the drug’s potential to treat cancer in patients Historically less than 15%.
What motivates people to participate in clinical trials of experimental drugs before efficacy or dosage is known?
What motivates patients to participate in clinical trials?
in one studya survey asking just this question was mailed to about 300 cancer trial participants. % said they were motivated by the possibility of obtaining medical benefit from treatment.
The next highest motivations for adults to participate in phase 1 trials were to trust investigators (17%), to stay hopeful (15%) and to help future patients (4%). This last category is considered medical altruism in the sense that they want to help advance medical science. These tend to be more common in patients with a favorable prognosis.
So why did nearly half of these patients enroll in cancer clinical trials in the hope of an effective drug, when the sole purpose of the trial was to find the right dosage?
Imagine you are my patient with leukemia that continues to burn despite multiple rounds of chemotherapy. My doctor told me that there is no better treatment, so I should go to a nearby university hospital or a large academic hospital that may be conducting clinical trials of new drugs.
Communication gaps when signing up for clinical trials
For people like my patient, who are fit enough to drive six hours to see a doctor, a clinical trial is the next step in treatment. My doctor told me there was a clinical trial, regardless of what the trial entailed or the promise that the drug might work. .
But perhaps the more common reason is that those of us involved in clinical research have not effectively communicated the true goals of such early-stage trials.
Such ineffective communication can lead to therapeutic misunderstandings. That is, the belief that the purpose of research is to directly benefit the individual patients enrolled in the trial. In reality, only prospective patients can benefit from research.
another study investigated how physicians communicated the risks and benefits of participating in a phase 1 trial to 85 families of childhood cancer patients. In 81 of 85 families, drug treatment risks were discussed 95% of the time. It’s a little surprising that this didn’t happen 100% of the time, because those phase 1 trials included chemotherapy.
Therapeutic benefit was discussed with approximately equal frequency in 75 of 85 families, 88% of the time. Altruism was mentioned in her 41% of family members. However, 13% of the conversations described the clinical trial as actually building a bridge to another treatment or extending life. There was no evidence that these first-person drug treatments were even effective in shrinking cancer.
“I’m not ready to throw in the towel yet.”
Terminally ill cancer patients seek treatment for a variety of reasons and are willing to endure significant side effects from drugs for the prospect of modest benefits.
As healthcare providers, it is our responsibility to understand these motivations and ensure that patients do not enroll in clinical trials with the wrong goals in mind. Also, special care must be taken not to misinform the patient about what the goals of treatment are. Possible.
I asked the patient if he was sure he wanted to pursue another treatment for his leukemia.
“No Doc, I’m not ready to throw in the towel yet. I’m still tough as nails!”
He qualified for the trial and spent the next month in the hospital, enduring considerable side effects from the treatment, all the while retaining his fine spirit. But at the end of the month, despite his and our efforts, the leukemia remained.
I saw him again at my clinic before he went home and apologized for how he had spent what turned out to be his last week. I felt terrible that I spent this time in a foreign city, enduring the blows of experimental treatments instead of going home.
he shook me off “Doctor, was it worth it? Maybe you’ll learn something by studying me and I can help someone in the future.”
His altruism was very touching. And maybe for my patients, it was just the point all along to get Moxie to take that step of treatment—to get in the ring again and say he’s explored every avenue.
Mikkael A. Sekeres, MD, MS is Chief of Hematology at the University of Miami Sylvester Comprehensive Cancer Center. He is a former chairman of the FDA’s Oncology Drug Advisory Committee and author of the book. Drugs and FDA: Safety, Efficacy, and Public ConfidenceFollow him on Twitter @MikkaelSekeres.